On May 6, 2021 Isofol Medical AB (publ) ("Isofol"), (Nasdaq First North Premier Growth Market: ISOFOL) reported its primary recruitment objective with the recruitment of 440 patients in the global phase III AGENT study (Press release, Isofol Medical, MAY 6, 2021, View Source [SID1234579419]). Today the company announces that it has completed the recruitment of Japanese patients in accordance with the regulatory requirements by the PMDA (the Japanese Medicines Agency) to reach market approval in Japan. As previously communicated, Isofol expects the top line results for the AGENT study to be available during H1 2022.

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Following today’s announcement of the completion of recruitment of Japanese patients, the full patient population, including the entire Japanese cohort, will be included in PMDA’s assessment for a potential market approval in Japan. For a market approval in Japan, the PMDA set a specific requirement for the number of participating Japanese patients of a total of 56 Japanese patients (of which 14 Japanese patients were already included in the primary recruitment of 440 patients) in the AGENT study. The rationale for the specific requirement from PMDA is e.g. that the metabolism of Japanese patients tends to differ from patients in other countries, which is why the effect and potential side effects must be investigated separately.

For the rest of the world, the original 440 patients will be analyzed for efficacy, but the full patient population will be analyzed for safety purposes.

"I am satisfied that we now have completed the recruitment of the Japanese patients, an important step on the way to receive market approval in Japan, the second largest oncology market worldwide. We are now looking forward to continue working with Solasia on the development and registration of arfolitixorin to bring a new treatment option to patients living with mCRC in Japan", said Ulf Jungnelius, M.D, CEO of Isofol.

Solasia Pharma ("Solasia") will fund and supervise clinical development activities in Japan and will be responsible for registrational filing, and following potential regulatory approvals, Solasia will, as the Market Authorization holder, be responsible for the commercialization of arfolitixorin in Japan. Isofol remain the global sponsor of the AGENT study.

"We are very pleased to have completed the recruitment of the target number of patients in Japan in the AGENT study and contributed to the global development of arfolitixorin. I would like to thank all the patients and investigators participating in the study, the CRO in charge of conducting the study, and Isofol, our partner and the sponsor of the AGENT study, for supporting us achieve this important goal. Patient recruitment was completed earlier than expected, and Solasia, together with Isofol, will further proceed development of arfolitixorin for market approval in Japan with the aim of becoming a new treatment option for mCRC patients", said Yoshihiro Arai, President & CEO of Solasia.

Arfolitixorin is evaluated in the AGENT study for the treatment of patients with first-line metastatic colorectal cancer (mCRC). The study is currently being conducted in the U.S., Canada, Europe, Australia and Japan in more than 90 clinics.

The information was submitted for publication, through the agency of the contact person set out above, at 08:30 CET on May 6, 2021.

About the AGENT study

The Phase III AGENT study is a randomized, controlled, multi-centre study assessing the efficacy and safety of arfolitixorin, [6R]-5,10-methylene-THF acid (MTHF), compared to leucovorin, both used in combination with 5- FU, oxaliplatin, and bevacizumab, in first-line metastatic colorectal cancer patients. Patients are randomized in a 1:1 ratio and the primary endpoint is overall response rate (ORR). The key secondary endpoints are progression free survival (PFS) and duration of response (DOR). Other secondary endpoints include overall survival (OS), number of curative metastasis resections, safety, and patient reported outcomes such as quality of life (QoL). Exploratory endpoints include pharmacokinetic (PK) measurements and level of gene expression of folate relevant genes in tumour cells. The study is designed to show superiority for arfolitixorin over leucovorin. The study is ongoing at approximately 90 sites in the U.S., Canada, Europe, Australia and Japan. In December 2020, the last of the AGENT study’s 440 patients was recruited, which is the basis in the statistical analysis plan. Recruitment has since continued in Japan to reach 56 Japanese patients. Isofol is now focusing on completing the ongoing AGENT study where the patients receive first-line standard treatment for metastatic colorectal cancer (mCRC). The company expects that the results of the AGENT study will be available during H1 2022.Further information about the study, including patient eligibility requirements, is available at www.clinicaltrials.gov id: NCT03750786.

About arfolitixorin

Arfolitixorin is Isofol’s proprietary drug candidate being developed to increase the efficacy of standard of care chemotherapy for advanced colorectal cancer. The drug candidate is currently being studied in a global Phase III study, AGENT. As the key active metabolite of the widely used folate-based drugs, arfolitixorin can potentially benefit more patients with advanced colorectal cancer, as it does not require complicated metabolic activation to become effective.

On May 6, 2021 Bio-Techne Corporation (NASDAQ: TECH) reported that its Board of Directors has decided to pay a dividend of $0.32 per share for the quarter ended March 31, 2021 (Press release, Bio-Techne, MAY 6, 2021, View Source [SID1234579265]). The quarterly dividend will be payable May 28, 2021 to all common shareholders of record on May 17, 2021. Future cash dividends will be considered by the Board of Directors on a quarterly basis.

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On May 6, 2021 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor-infiltrating lymphocyte, TIL, and peripheral-blood lymphocyte, PBL), reported first quarter 2021 financial results and corporate updates (Press release, Iovance Biotherapeutics, MAY 6, 2021, View Source [SID1234579337]).

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Maria Fardis, Ph.D., President and Chief Executive Officer of Iovance, stated, "During the first quarter of 2021 we continued to work on our potency assays in support of a BLA for lifileucel, which is our top priority at Iovance. We continued developing multiple assays in parallel and submitted additional potency assay data to the FDA. I am also very pleased to highlight the strength of clinical data for TIL being presented at the recent and upcoming medical meetings and journal publications, including the first clinical data for TIL in combination with pembrolizumab in melanoma at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting. We believe durable responses for Iovance TIL in relapsed or refractory metastatic melanoma, paired with data to be presented for TIL in combination with anti-PD1 therapy in earlier treatment settings, solidify the broader potential for Iovance TIL and further demonstrate our leadership in the development, manufacturing, and potential commercialization of TIL cell therapy."

First Quarter 2021 Highlights and Recent Corporate Updates

Clinical:

TIL therapy, lifileucel, in melanoma: Updated data from Cohort 2 in the C-144-01 study of lifileucel in advanced melanoma were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2021 Annual Meeting. As of the December 2020 data cutoff for the presentation, lifileucel showed a 36.4% overall response rate (4.5% complete responses and 31.8% partial responses); median duration of response (DOR) was not reached at 28.1 months of median study follow up as assessed by investigators (n=66). Available care for Cohort 2 patients is chemotherapy, with an overall response rate of four to 10 percent and overall survival of only seven to eight months. Updated Cohort 2 data has been accepted for an oral presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. A manuscript of the Cohort 2 data has also been accepted for a forthcoming publication in a high impact oncology journal.
TIL therapy, lifileucel, in cervical cancer: The C-145-04 study of lifileucel, formerly LN-145, is intended to support a BLA submission for metastatic cervical cancer. Inclusion of both pivotal cohort 1 (post-chemotherapy) and cohort 2 (post-anti-PD-1/PDL-1) in the BLA may strengthen the potential label and reflect the expected upcoming treatment landscape in cervical cancer. Patient dosing is complete in both Cohorts 1 and 2, and enrollment continues in Cohort 3 (lifileucel in combination with pembrolizumab in cervical cancer patients who are naïve to anti-PD-1 therapy).
TIL therapy in non-small cell lung cancer (NSCLC): Iovance has activated 10 U.S. clinical sites and patients have consented in the potential registration-directed study, IOV-LUN-202, to investigate LN-145 in patients with recurrent or metastatic NSCLC, without driver mutations, who previously received a single line of approved systemic therapy (combined checkpoint inhibitor (CPI) plus chemotherapy). The company also continues to investigate LN-145 in several additional NSCLC populations with unmet need across three cohorts in the IOV-COM-202 basket study.
TIL therapy in combination with pembrolizumab in melanoma and head and neck squamous cell carcinoma (HNSCC): Iovance is investigating TIL in combination with pembrolizumab in earlier treatment settings in melanoma and HNSCC. Initial data from Cohort 1A in the IOV-COM-202 study, which is evaluating lifileucel in combination with pembrolizumab in melanoma, will be presented at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting. Patients with HNSCC continue to enroll in the expanded Cohort 2A in the IOV-COM-202 study to receive LN-145 in combination with pembrolizumab.
TIL clinical program updates: To date, over 450 patients have been dosed with Iovance TIL products with more than a 90 percent manufacturing success rate.
Regulatory

Potency assays for lifileucel: Iovance recently submitted additional potency assay data to the FDA, while continuing to evaluate additional assays as backup options in support of the BLA. Submission of the BLA is anticipated during 2021. Expeditious resolution of the potency assay for lifileucel in melanoma is also a key step towards BLA submission plans in cervical cancer.
Manufacturing:

Iovance Cell Therapy Center (iCTC): Activities at the iCTC have commenced to support start of clinical manufacturing in late 2021. The expected commercial manufacturing remains on track to start in 2022.
Generation 3 (Gen 3) manufacturing: A shorter 16-day third generation manufacturing process is being explored in a cohort of metastatic melanoma patients in the IOV-COM-202, where patient dosing has initiated using Gen 3. In addition, a cohort of NSCLC patients in the IOV-LUN-202 study will receive product manufactured by this Gen 3 process.
Corporate:

Cash position of $610.2 million on March 31, 2021 is expected to be sufficient into 2023 to deliver on pipeline programs.
A strong organization of more than 250 employees, of which 76 percent have more than a year of cell therapy experience, is in place to advance research, development, manufacturing, and commercial launch preparations.
On May 6, 2021, Iovance entered into an amended license agreement with the National Institutes of Health (NIH), which adds additional exclusive, worldwide patent rights in certain indications to cytokine-tethered TIL technology and expands the non-exclusive, worldwide field of use to all cancers.
Iovance continues to expand its intellectual property portfolio and currently owns more than 25 granted or allowed U.S. and international patents for compositions and methods of treatment in a broad range of cancers relating to the Gen 2 manufacturing process. Iovance’s Gen 2 patent rights are expected to provide exclusivity through 2038. Iovance’s portfolio also includes patent applications and granted patents directed towards Gen 3 manufacturing, selected TIL products, stable and transient genetic TIL modifications, tumor digest and fragment compositions and methods (including cryopreservation), and combinations of checkpoint inhibitors and TIL products.
First Quarter 2021 Financial Results

Iovance held $610.2 million in cash, cash equivalents, investments and restricted cash at March 31, 2021 compared to $635.0 million at December 31, 2020. The company anticipates that the year-end balance of cash, cash equivalents, investments and restricted cash will be sufficient into 2023.

Jean-Marc Bellemin, Chief Financial Officer, stated, "The continued strength of our balance sheet puts Iovance in an excellent position to fulfill our operating plan and advance our pipeline. We are judicious in our investments to maximize value and deliver on our commitments for patients and our shareholders."

Net loss for the first quarter ended March 31, 2021, was $75.4 million, or $0.51 per share, compared to a net loss of $69.6 million, or $0.55 per share, for the first quarter ended March 31, 2020.

Research and development expenses were $55.9 million for the first quarter ended March 31, 2021, a decrease of $1.0 million compared to $57.0 million for the first quarter ended March 31, 2020.

The decrease in research and development expenses in the first quarter 2020 over the prior year period was primarily attributable to a decrease in manufacturing and clinical costs following the completion of enrollment in the pivotal cohorts for melanoma and cervical cancer.

General and administrative expenses were $19.6 million for the first quarter ended March 31, 2021, an increase of $5.8 million compared to $13.9 million for the first quarter ended March 31, 2020.

The increases in general and administrative expenses in the first quarter 2021 compared to the prior year period was primarily attributable to growth of the internal general and administrative team and higher stock-based compensation expenses.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss the first quarter 2021 financial results and corporate updates. The conference call dial-in numbers are 1-800-773-2954 (domestic) or 1-847-413-3731 (international) and the access code is 50155289. The live webcast can be accessed in the Investors section of the company’s website at View Source The archived webcast will be available for a year in the Investors section at www.iovance.com.

On May 6, 2021 Caladrius Biosciences, Inc. (Nasdaq: CLBS) ("Caladrius" or the "Company"), a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse disease, reported financial results for the three months ended March 31, 2021 (Press release, Caladrius Biosciences, MAY 6, 2021, View Source [SID1234579353]).

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"We are at a truly exciting point in our evolution with tremendous opportunities ahead of us. While the pandemic has impacted many companies, during the first quarter of 2021 we were able to both markedly strengthen our financial position and advance and expand our clinical pipeline," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. To date, we are seeing good progress with site activation for our Phase 2b clinical trial of CLBS16 in the U.S., known as the FREEDOM Trial, for the treatment of coronary microvascular dysfunction as we continue to accelerate enrollment. Additionally, we remain optimistic that we soon will complete enrollment in our registration-eligible study of HONEDRA in critical limb ischemia and Buerger’s disease in Japan. However, enrollment for this program has been greatly impacted by the Japanese government-issued states of emergency tied to the pandemic. Lastly, we are working with the U.S. Food and Drug Administration ("FDA") to finalize the protocol design for our CLBS201 proof-of-concept study in diabetic kidney disease and have targeted initiation of that Phase 2 study in the third quarter of 2021."

Product Development and Financing Highlights

CLBS16 for the treatment of coronary microvascular dysfunction

Caladrius reported in May 2020 the compelling positive results of its ESCaPE-CMD Phase 2a study of CLBS16 for the treatment of coronary microvascular dysfunction ("CMD"), a disease that continues to be underdiagnosed and potentially afflicts millions annually – a vast majority of whom are female – with no current treatment options. The Company is committed to raising awareness of this growing women’s health crisis and finding an effective treatment. Caladrius recently initiated, and is currently treating patients in, a rigorous 105-subject Phase 2b clinical trial (the FREEDOM Trial), which to our knowledge, is the first controlled regenerative medicine trial in CMD. The trial is targeted to complete enrollment by the end of 2021 with top line data anticipated for the third quarter of 2022. This double-blind, randomized, placebo-controlled Phase 2b trial will evaluate the efficacy and safety of delivering autologous CD34+ cells to the hearts of subjects with CMD.

HONEDRA (CLBS12) for the treatment of critical limb ischemia

The Company’s open-label, registration-eligible study of SAKIGAKE-designated HONEDRA in Japan for the treatment of critical limb ischemia ("CLI") and Buerger’s disease (an orphan-sized subset of CLI) has shown strong results to date. The initial responses observed in the subjects who have reached an endpoint in this study are consistent with a therapeutic effect and safety profile reported by previously published clinical trials in Japan and the U.S. The study’s enrollment continues to be slowed by the pandemic’s impact in Japan, however, the Company is encouraged by the patient pre-screening pipeline and continues to make progress towards study completion, the exact date of which is impossible to predict given the continuing impact of COVID-19 on clinical trials in Japan. While the final outcome of the trial will depend on all data from all subjects, the data to date is very encouraging (~60% of subjects in the completed Buerger’s disease cohort have reached a positive "CLI-free" endpoint despite a natural history of such patients that predicts continuing disease progression to amputation). In the U.S., the Company was pleased to report that the FDA granted orphan designation to CLBS12 as a treatment for Buerger’s disease.

CLBS201 for the treatment of diabetic kidney disease

The Company’s most recently proposed development program, CLBS201, is designed to assess the safety and efficacy of CD34+ cell therapy as a treatment for diabetic kidney disease in patients not yet requiring dialysis. Based on a wealth of published preclinical and early clinical data, it appears that the innate ability of CD34+ cells to promote the growth of new microvasculature could be a means to attenuate the progression of the disease or even reverse the course of diabetic kidney disease. A Phase 2 proof of concept, randomized, placebo-controlled study is planned for initiation in the second half of 2021.

OLOGOTM for the treatment of no option refractory disabling angina ("NORDA")

Caladrius acquired the rights to data and regulatory filings for a CD34+ cell therapy program for NORDA that had been advanced to Phase 3 by a previous sponsor. Based on the clinical evidence from the completed studies that a single administration of OLOGOTM reduces mortality, improves angina and increases exercise capacity in patients with otherwise untreatable angina, this product received Regenerative Medicine Advanced Therapy ("RMAT") designation from the FDA. Caladrius remains in ongoing discussions with the FDA regarding the size and scope of an appropriate and practical Phase 3 trial, which in combination with previously filed Phase 1, 2 and 3 data, will be considered for the registration of OLOGOTM. Notably, the RMAT designation affords the product a 6-month review time for a biologics license application ("BLA"), once submitted.

Sufficient capital to fund operations beyond multiple key data readouts (>2023)

As previously disclosed, in January 2021, Caladrius raised $25.0 million in a private placement priced at-the-market under Nasdaq rules. In February 2021, the Company announced that it closed a $65.0 million capital raise through the sale of its common stock to several institutional and accredited investors in two registered direct offerings priced at-the-market under Nasdaq rules.

First Quarter 2021 Financial Summary

Research and development expenses for the three months ended March 31, 2021 were $5.1 million, compared to $1.5 million for the three months ended March 31, 2020. Research and development in the current year period focused on the advancement of our ischemic repair platform and related to:

•Ongoing expenses for HONEDRA in critical limb ischemia and Buerger’s disease in Japan for which we continue to focus spending on patient enrollment and Japanese NDA preparation; and

•Expenses associated with efforts to advance the FREEDOM Trial where the first patient was dosed in the first quarter of 2021; and

•Expenses associated with the planning and preparation of an IND and proof-of-concept protocol for CLBS201 as a treatment for diabetic kidney disease.

General and administrative expenses, which focus on general corporate related activities, were $3.0 million for the three months ended March 31, 2021 compared to $2.6 million for the three months ended March 31, 2020, representing an increase of 18%.

Overall, net losses were $8.1 million and $4.0 million for the years ended March 31, 2021 and 2020, respectively.
Balance Sheet Highlights

As of March 31, 2021, Caladrius had cash, cash equivalents and marketable securities of approximately $111.5 million. Based on existing programs and projections, the Company remains confident that its current cash balances will fund its operations for the next several years, notably through study completion for the FREEDOM Trial, through the registration-eligible study completion for HONEDRA and through the Phase 2 proof-of-concept study for CLBS201, while still providing capital to explore additional pipeline expansion opportunities.

Conference Call
Caladrius will hold a live conference call today, May 6, 2021, at 4:30 p.m. (ET) to discuss financial results, provide a business update and answer questions. To join the conference call, please refer to the dial-in information provided below. A live webcast of the call will also be available under "Events" in the Investors section of the Caladrius website, View Source, and will be available for replay for 90 days after the conclusion of the call.

On May 6, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported that it will report financial results and highlights for the quarter ended March 31, 2021 on Thursday, May 13, 2021, after the U.S. financial markets close (Press release, Regulus, MAY 6, 2021, View Source [SID1234579370]).

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The Company will host a conference call and live audio webcast on Thursday, May 13, 2021 at 5:00 pm Eastern Daylight Time to report its first quarter 2021 financial results and provide a corporate update. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 6947037. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 6947037. The webcast and telephone replay will be archived on the Company’s website at www.regulusrx.com following the call.